A new therapy for AML is necessary because, although there are several treatment options for
patients with AML, these treatments are very toxic and not available to all AML patients or
only useful for acute promyelocytic leukemia (APL). Also, there is essentially no treatment
for patients with refractory or relapsed AML outside of bone marrow transplant.
Accordingly, there is a great medical need for a safe and effective therapy for AML,
especially refractory and relapsed AML. Also, although hypomethylating agents have been
found to be effective against MDS, these agents are toxic. Furthermore, after relapsing from
a hypomethylating agent, there is no treatment for this disease.

A nearly completed clinical trial of CPI-613 (Cornerstone Study# CL-CPI-613-009 or Wake
Forest Study# CCCWFU 29109, under IND# 107,800) shows that CPI-613 is well tolerated at
doses as high as 3,000 mg/m2. Results from this nearly completed trial also suggest that
CPI- 613 may be effective against refractory and relapsed AML, as well as against MDS that
is relapsed from a hypomethylating agent. Therefore, CPI-613 may be a suitable treatment
option for refractory/relapsed AML and MDS relapsed from a hypomethylating agent. The
promising preliminary efficacy data from Study# CL-CPI-613-009 (Wake Forest Study# CCCWFU
29109, under IND# 107,800) is the basis on which Cornerstone is conducting the current Phase
2a trial to further assess the efficacy of CPI-613 against these diseases.

Inclusion Criteria:

- Have either documented refractory or relapsed AML, or documented MDS of any risk
group that has failed a hypomethylating agent (such as decitabine [Vidaza] and
azacitidine [AZA]). (Therapy failure with a hypomethylating agent is defined as
patients who have been sufficiently treated with hypomethylating agents without
response in the opinion of the treating physician, or whose disease has progressed or
relapsed while on a hypomethylating agent.) Has never been treated with CPI-613.

- Women of child-bearing potential (i.e., women who are pre-menopausal or not
surgically sterile) must use accepted contraceptive methods (abstinence, intrauterine
device [IUD], oral contraceptive, or double barrier device) during the study, and
must have a negative serum or urine pregnancy test within 1 week prior to treatment
initiation.

- Fertile men must practice effective contraceptive methods during the study, unless
documentation of infertility exists.

- No radiotherapy, surgery or hormonal therapy for any kind of within 2 weeks prior to
participating in this study. Patients must have fully recovered from the acute
toxicities of any prior treatment with any anti-cancer drugs (including
hypomethylating agents in MDS patients), radiotherapy or other anti-cancer modalities
(i.e., returned to baseline status as noted before most recent treatment) for any
tumors. Patients with persisting, stable chronic toxicities from such prior treatment
≤Grade 1 are eligible, but must be documented as such.

- Recombinant erythropoietin or G-CSF is not allowed, since CPI-613 does not induce
myelosuppression.

- No evidence of active or serious infection of any kind within the past month. No
systemic fungal, bacterial, viral or other infection not controlled, defined as
exhibiting ongoing signs/symptoms related to the infection and without improvement,
despite appropriate antibiotics or other treatment.

- Patients receiving any standard or investigational therapy for any tumor indication
within the past 2 weeks, or any investigational agent for any indication within the
past 4 weeks, prior to the study.

- Patients who have received immunotherapy of any type for any indications within the
past 4 weeks prior to the study.

- Ongoing oral corticosteroids are not permitted. However, topical and inhaled
corticosteroids are permitted, and prophylactic steroids are allowed for transfusion
reactions.

- Life expectancy less than 2 months.

- Pregnant women, or women of child-bearing potential not using reliable means of
contraception.

- Lactating females.

- Fertile men unwilling to practice contraceptive methods during the study period.

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